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National strategy in the works on ‘orphan drugs’

After years of debate, Canada is set to create a national framework to improve access to "orphan drugs" for people who suffer from rare disorders.

"Orphan drugs" is a term applied to medications that are used to treat chronic or debilitating conditions that only affect a handful of people.

The new framework, announced by federal Health Minister Leona Aglukkaq, will provide a national strategy for authorizing and monitoring the use of orphan drugs as well as helping to spur research and development of new drugs to treat rare diseases. Aglukkaq also announced that Canada will launch Orphanet, a site dedicated to improving diagnosis, care and treatment of rare diseases.

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"It's huge news," said Maureen Smith, secretary of the Canadian Organization for Rare Disorders (CORD), who has a rare disease called congenital panhypopituitarism. "We've been waiting for this for a very long time."

Orphan drugs are typically expensive and difficult to research and bring to market, given the small patient population in which they can be studied. CORD hopes the framework will improve access and encourage more provinces to cover the costs. Some provinces, such as British Columbia, Alberta and Ontario, have significantly improved access and funding in recent years. But others have operated with a patchwork system, Smith said.

Chaim Bell, associate professor of medicine at the University of Toronto and physician and scientist at Mount Sinai Hospital, cautioned that because of the small patient pool, orphan drugs may be approved with limited evidence, or unanswered questions about side effects. He said Health Canada should establish a registry to track adverse side effects, so doctors develop a better understanding of the potential risks.

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